Alliance executives often struggle with measuring the value delivered by alliances and the alliance management function. Alnylam CEO John Maraganorehas no such difficulty. His company is a pioneer in the development of RNA interference (RNAi) therapies. Amidst great fanfare preceding the JP MorganHealthcare Conference, in January of this year Alnylam signed a far-reaching partnership deal with Sanofi-owned Genzyme. In his lively and richly detailed keynote address at the 2014 ASAP BioPharma Conference in Boston USA on Thursday, Sept. 4, Maraganore clearly articulated this alliance’s growth and financial implications for Alnylam business.

“This provided a major capital infusion as well as expense sharing going-forward,” he explained in a talk entitled “Alnylam-Genzyme: Breakthrough Therapy Made Possible by Breakthrough Partnership. “Our deal with Genzyme took our balance sheet from $325 million to over $1 billion. It transformed our balance sheet, giving us financial independence all the way to becoming profitable.”

Yet this financial payoff still isn’t what Maraganore considers the most significant value of this alliance. “More than the money, we were convinced that working with Genzyme would increase the pie,” he said. “Without this deal, we probably would have been more stepwise in building our pipelines.”

RNAi therapeutics, Maraganore explained to a rapt audience of biopharma industry alliance executives, are a new class of innovative medicines based on a clinically validated platform that harnesses natural pathways for therapeutic gene silencing. “Hundreds of diseases can be knocked down with our strategy—the ‘Alnylam 5x15 Strategy’—which provides a reproducible and modular path for genetic medicines.”

Maraganore’s talk began with an overview of the science that brought Genzyme and Alnylam together.

“What we do, and why it’s so potentially powerful, is we change nothing but the sequencing of the drug to make it attack a different gene. In part one of our development, we’ve figured out the targeting of liver-expressed genes. You know with almost complete certainty that you will impact by targeting that gene,” he explained. “The second part of our strategy helps to stack the odds in our favor. If we can learn in Phase One trials that our drug is working, and if we can learn in that study how we get to the right dose regimen, we get through another critical unknown. A lot of drugs fail in Phase Three because you don’t get to the right dose.”

Maraganore frankly acknowledged that the final part of Alnylam’s strategy—getting through regulatory approval and on the market—“has no free lunch.” However, the company’s product strategy has allowed it to reduce the scope and cost of Phase Three trials.

“We really focused on areas with a definable path to approval and market … where our Phase Three program would be comprised of hundreds of patients as compared to thousands or tens of thousands of patients. We’re a small company, so our strategy has led us into the rare disease space,” he explained, emphasizing that Alnylam has taken a “collaborative approach with patients, payers, and regulators.”

Even just with an initial focus on the liver, Alnylam has no shortage of diseases to target—“almost everything in genetic disease space,” Maraganore said. Current examples include:

Hemophilia. “Why would knockdown strategy work if caused by a genetic lack? By knocking down anticoagulants,” Maraganore said. “It’s a $9 billion market with high unmet need.”

Porphyria, an ultra-rare orphan disease.

In ASAP Media’s next blog post on Maraganore’s speech, we will delve into the Alnylam CEO’s fascinating review of his company’s groundbreaking partnership with Genzyme—what he described as “a transformational alliance to expand and accelerate global product value.”